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- Consensus guidelines for the diagnosis and clinical management of Erdheim-Chester disease (2014) PubMed
All lines of therapy
Sirolimus & Prednisone
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|Gianfreda et al. 2015||Pilot, <20 pts in this arm|
- Sirolimus (Rapamune) 2 mg PO once per day, titrated to achieve a blood level of 8 to 12 ng/mL
- Prednisone (Sterapred) as follows:
- Month 1: 0.75 mg/kg/day PO
- Month 2: 0.5 mg/k/day PO
- Months 3 & 4: 0.25 mg/kg/day PO
- Months 5 & 6: 0.125 mg/kg/day PO
- Month 7 onwards: tapered to a dose of 2.5 to 5 mg PO once per day
Given for up to 2 years for responders and stable disease, with optional extension beyond 2 years
- Gianfreda D, Nicastro M, Galetti M, Alberici F, Corradi D, Becchi G, Baldari G, De Filippo M, Ferretti S, Moroni G, Foti R, Di Gangi M, Jeannin G, Saffroy R, Emile JF, Buzio C, Vaglio A. Sirolimus plus prednisone for Erdheim-Chester disease: an open-label trial. Blood. 2015 Sep 3;126(10):1163-71. Epub 2015 Jun 3. link to original article contains verified protocol PubMed
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|Hyman et al. 2015||Phase II, <20 pts in this arm|
This was part of a basket trial, all patients had BRAF V600E mutations.
- Vemurafenib (Zelboraf) 960 mg PO BID
Duration of treatment not specified
|Haroche et al. 2014||Pilot, <20 pts|
Patients enrolled all had BRAF V600E mutation. This is the dose after de-escalation after the first four patients had excess adverse events.
- Vemurafenib (Zelboraf) 480 mg PO BID
Given until progression of disease or unacceptable toxicity
- Haroche J, Cohen-Aubart F, Emile JF, Maksud P, Drier A, Tolédano D, Barete S, Charlotte F, Cluzel P, Donadieu J, Benameur N, Grenier PA, Besnard S, Ory JP, Lifermann F, Idbaih A, Granel B, Graffin B, Hervier B, Arnaud L, Amoura Z. Reproducible and sustained efficacy of targeted therapy with vemurafenib in patients with BRAFV600E-mutated Erdheim-Chester disease. J Clin Oncol. 2015 Feb 10;33(5):411-8. Epub 2014 Nov 24. link to original article contains verified protocol PubMed
- Hyman DM, Puzanov I, Subbiah V, Faris JE, Chau I, Blay JY, Wolf J, Raje NS, Diamond EL, Hollebecque A, Gervais R, Elez-Fernandez ME, Italiano A, Hofheinz RD, Hidalgo M, Chan E, Schuler M, Lasserre SF, Makrutzki M, Sirzen F, Veronese ML, Tabernero J, Baselga J. Vemurafenib in multiple nonmelanoma cancers with BRAF V600 mutations. N Engl J Med. 2015 Aug 20;373(8):726-736. link to original article contains protocol link to PMC article PubMed